Neurofibromatosis Network Advocacy Program NF Network Advocacy - PowerPoint PPT Presentation

Neurofibromatosis Network Advocacy Program NF Network Advocacy Program Structure your Congressional meeting

Structure Your Congressional Meeting • Introduction & Thank you • Explain NF • Impact • Personal Story • Military Benefit • How NF Impacts State/District • Explain the NF Research Program (NFRP) • How the NFRP and NIH complement each other • How Congress can help NF Network Advocacy Program 2017 Advocacy Program

What is Neurofibromatosis (NF)? • Highly variable genetic disorder of the nervous system which can affect every organ system • Causes tumor growth along nerves • NF is a family of tumor disorders where a protein is lacking • Without this protein, tumors can grow in the brain, spine and along nerves that lead to a variety of issues • NF occurs worldwide in all races and ethnic groups and both sexes and can appear in any family • Some tumors may be visible, and some may not • NF affects more than 100,000 Americans; this makes NF more prevalent than Cystic Fibrosis, hereditary Muscular Dystrophy, Huntington’s disease and Tach Sachs combined NF Network Advocacy Program 2017 Advocacy Program

Neurofibromatosis Manifestations Nervous System Disorders Tumors Neurofibromas Growing Along Nerves Epilepsy • Including Skin • Brain Headaches • Spinal Cord Malignancies Visual Impairments Chronic Myeloid Leukemia Tumors of the eye Malignant peripheral nerve Blindness sheath tumor (MPNST) Retinal hamartomas Learning Deficits/ Deafness Cognitive Disorders Tumors of the ear Learning Disabilities ADHD Autism Vascular Disease Motor Deficits Hypertension Dysplasia of blood vessels Skin Conditions Café-au-lait spots Musculoskeletal Disorders Dermal neurofibromas Muscle weakness Scoliosis Severe Pain Bone Abnormalities Congenital hydrocephalus

Personal Story Tell your personal story (the most important part of the meeting!) Insert your personal story and photos 2017 Advocacy Program

Military Benefit

Impact on State/District US population: 313,800,000 US NF population: 128,032 • How NF impacts State/District • Research funding to state

How is NF research funded? • Through the Department of Defense and several Institutes at the National Institutes of Health (NIH). 2017 Advocacy Program

Neurofibromatosis Research Program (NFRP) • The Army’s Neurofibromatosis Research Program was created in 1996 and is one of the Congressionally Directed Medical Research Programs (CDMRP). • The NFRP is an efficiently run national program that offers cutting edge awards through a competitive peer-review process to fill gaps in ongoing research, complementing initiatives sponsored by other agencies, such as the National Institutes of Health (NIH) • The NFRP has participation from the NF researchers, NIH and from NF affected individuals. • The NF Clinical Trials Consortium, established with NFRP funds in 2006, significantly accelerates the clinical trial process by recruiting patients from 21 clinical sites with an operational center to analyze the data. It has processed 9 clinical trials with an additional 4 trials opening in 2017. 2017 Advocacy Program

National Institutes of Health (NIH) • NF research is conducted by several institutes at the NIH • NF specific report language encourages increased funding for NF research at NIH NFRP and NIH research programs collaborate • Due to the success of the NFRP, NF Research at NIH has proportionally increased since the inception of the DoD program. • The NFRP funds cutting edge higher risk research projects, these projects collect data which increases the chance for good NF science projects to be funded by NIH. • NIH and NFRP collaborate on NF research through out the year. • NIH holds an inter institute meeting – NFRP is represented • NFRP review panels – NIH is represented . 2017 Advocacy Program

Success of the Neurofibromatosis Research Program • Preclinical studies funded by the NFRP lead to data used to create the successful study done by, Brigitte Widemann, M.D. from National Cancer Institute • Recent paper published in the New England Journal of Medicine on Dec. 29, 2016 • Shows that Selumetinib shrinks plexiform tumors by 20 % in 70 percent of enrolled patients. • Evidence of Clinical improvement, decrease in tumor-related pain, improvement in motor function, and decreased disfigurement was reported Philip Moss from Birmingham, Alabama has had a 36% reduction in the size of his tumor. Philips Mom says, “We rarely hear children ask Philip what is wrong with his neck anymore.”

FY 2017 & FY 2018 Request • Defense Appropriation Request: $15 million in funding for the Army’s NF Research Program (NFRP) in the FY 2017 and FY2018 Department of Defense Appropriations bills. • Labor HHS Appropriations Request: Support inclusion of report language on NF research at the National Institutes of Health be included in the FY 2018 Labor, Health and Human Services, Education Appropriations bill. 2017 Advocacy Program

Request How you can help: • Sign onto the Dear Colleague letter that will be circulated in both the House and Senate. We will contact you when it is circulated. • Letters sponsors include: • Rep. Luis Gutierrez (D-IL) – Adrienne Castro • Rep. Glenn Grothman (R-WI) – Vincent Giglierano • Sen. Edward Markey (D-MA) • Include these requests on your priority/wish list and submit the request to the Defense Appropriations Subcommittee ($15 million) and the Labor, Health and Human Services Appropriations Subcommittee (report language). 2017 Advocacy Program

Thank you for your time!